๐Ÿ›๏ธ Verified NIH Record โ€ข Updated 2025-01-29

A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY) (NCT04853576)

A Phase 1/2 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous Clustered Regularly Interspaced Short Palindromic Repeats Gene-edited CD34+ Human Hematopoietic Stem and Progenitor Cells (EDIT-301) in Subjects With Severe Sickle Cell Disease

๐Ÿ”ต Active, Not Recruiting Phase1 / Phase2 ๐Ÿ“‹ Clinical Research Protocol Sponsor: Editas Medicine, Inc.

๐Ÿฉบ Plain-English Summary (8th-Grade Level)

The purpose of this study is to evaluate the efficacy, safety and tolerability of treatment with EDIT-301 in adult and adolescent participants with severe sickle cell disease (SCD).

Who can join: Review the eligibility criteria below.

Location: UCSF Benioff Children's Hospital โ€” Oakland, California

Age range: 12 Years to 50 Years

๐Ÿงช Interventions in This Study

  • EDIT-301 (GENETIC)

๐Ÿ“‹ "Do I Qualify?" โ€” 1-Minute Self Screener

Instant Checklist

Full eligibility criteria are available on the official NIH protocol. View the criteria โ†—

๐Ÿ“ All Participating Trial Locations (24 Sites)

  • UCSF Benioff Children's Hospital โ€” Oakland, California
  • Children's Hospital Colorado โ€” Aurora, Colorado
  • Smilow Cancer Hospital โ€” New Haven, Connecticut
  • Johns Hopkins All Children's Hospital โ€” St. Petersburg, Florida
  • Children's Healthcare of Atlanta โ€” Atlanta, Georgia
  • Ann & Robert H. Lurie Children's Hospital of Chicago โ€” Chicago, Illinois
  • University of Mississippi Medical Center โ€” Jackson, Mississippi
  • Hackensack University Medical Center โ€” Hackensack, New Jersey
  • Columbia University Medical Center - Department of Pediatrics โ€” New York, New York
  • Columbia University Medical Center โ€” New York, New York
  • The University of North Carolina at Chapel Hill โ€” Chapel Hill, North Carolina
  • Atrium Health โ€” Charlotte, North Carolina
  • University Hospitals Rainbow Babies & Children's Hospital โ€” Cleveland, Ohio
  • Cleveland Clinic โ€” Cleveland, Ohio
  • Nationwide Children's Hospital โ€” Columbus, Ohio
  • The James Cancer Hospital โ€” Columbus, Ohio
  • Children's Hospital of Philadelphia โ€” Philadelphia, Pennsylvania
  • Medical University of South Carolina โ€” Charleston, South Carolina
  • Tristar Medical Group Children's Specialists/Sarah Cannon Center for Blood Cancers โ€” Nashville, Tennessee
  • Texas Oncology - Baylor Charles A. Sammons Cancer Center โ€” Dallas, Texas
  • Cook Children's โ€” Fort Worth, Texas
  • Ottawa Hospital Research Institute โ€” Ottawa, Ontario
  • Princess Margaret Cancer Centre โ€” Toronto, Ontario
  • Centre Hospitalier Universitaire Sainte-Justine โ€” Montreal, Quebec

๐Ÿ“ž Contact the Study Coordinator

Phone: (877) 827-3222

Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.

๐Ÿ’ฐ Cost, Insurance & Patient Rights

  • 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
  • Routine Patient Care: Under federal law (ACA ยง 2709) and many state statutes, routine care costs are covered during participation.
  • Voluntary Participation: You may withdraw at any time without affecting your standard medical care.

๐Ÿ’ก How to Participate

  1. Check the checklist above to see if you may qualify.
  2. Contact the study coordinator using the phone/email above.
  3. Ask about the visit schedule, what is covered at no cost, and any travel support.
  4. Confirm with your own doctor before making a decision.
๐Ÿ›๏ธ View Official NIH Record โ†—